Gene therapy for p47 phox form of CGD

The CGD Society are delighted that researchers from the Institute of Child Health at University College London have received funding to develop gene therapy for the second most common form of CGD - p47 phox deficiency.  Around one in four children with CGD have this type.

Action Medical Research awarded the grant of £80,249 over 18 months to Professor Adrian Thrasher and Dr Giorgia Santilli. The project will concentrate on the laboratory stages of gene therapy work and will build on knowledge previously gained by designing gene therapy for X-CGD, which is now at the clinical trial stage.

Dr Caroline Johnston, Research Evaluation Manager at Action Medical Research says: "Children with CGD have faults in the gene for a protein that helps immune cells fight infection. The new therapy will work by inserting a corrected copy of that gene into children's immune cells. It is hoped the new gene therapy will prove life-changing for children with CGD."

Related link http://www.action.org.uk/our-research/developing-new-treatment-children-rare-disorder-who-are-vulnerable-life-threatening-inf